Orchard Therapeutics Secures Partnerships to Broaden Access to LibmeldyTM for Eligible Patients in Middle East & Turkey
Orchard Therapeutics (Nasdaq: ORTX) is expanding its commercial presence in the Middle East and Turkey through exclusive collaborations with GenPharm Services and GEN. These partnerships aim to enhance access to Libmeldy, the first gene therapy approved for early-onset metachromatic leukodystrophy (MLD), which had EU approval in December 2020. The agreements facilitate treatment access for patients in seven additional markets: Saudi Arabia, Kuwait, UAE, Qatar, Bahrain, Oman, and Turkey. This initiative is crucial, as MLD is more prevalent in these regions.
- Expansion of commercial reach in the Middle East and Turkey for Libmeldy.
- Collaboration with established regional partners GenPharm Services and GEN.
- Access to treatment for MLD patients in seven new markets.
- None.
BOSTON and LONDON, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced plans to extend the company’s commercial reach in the Middle East and Turkey through exclusive agreements with GenPharm Services and GEN, two leading regional specialty pharmaceutical companies with extensive experience in rare genetic disease. GenPharm and GEN will collaborate with Orchard to facilitate access to treatment with Libmeldy in European-based qualified treatment centers for eligible patients living in the Middle East and Turkey.
Libmeldy was approved in the EU in December 2020 as the first gene therapy for the treatment of early-onset metachromatic leukodystrophy (MLD), an inherited, neurodegenerative disorder. Patients from the Middle East and Turkey were among those who participated in the clinical studies and/or compassionate use programs that supported the marketing authorization for Libmeldy in the EU.
“MLD is a devastating disease found at a higher rate in Middle Eastern and Turkish populations than those in the U.S. and Europe, which heightens our urgency to establish access mechanisms for Libmeldy in support of eligible patients in these regions,” said Frank Thomas, president and chief operating officer, Orchard Therapeutics. “Orchard’s agreements with GenPharm and GEN represent an innovative way to scale through collaboration, leveraging the pre-existing infrastructure, expertise and relationships of our partners to quickly and efficiently extend our reach to serve patients in these areas of the world.”
Under terms of the agreements, GenPharm and GEN will be responsible for all activities related to identifying patients, driving disease awareness, and ensuring market access for Libmeldy in their respective territories. Combined, these partnerships will expand access to Libmeldy for patients living in the following seven additional markets: Saudi Arabia, Kuwait, UAE, Qatar, Bahrain, Oman and Turkey.
“We launched GenPharm Services nearly nine years ago to address significant gaps we saw in meeting rare disease patients’ needs in the Middle East region,” said Karim Smaira, co-founder of GenPharm Services. “Given the severity of MLD and the lack of any other approved treatment options, we are honored to partner with Orchard Therapeutics to support eligible young MLD patients and their families in getting to Europe for treatment with such an innovative and promising therapy as Libmeldy.”
“As a leading specialty pharmaceutical company in Turkey, GEN is excited by the partnership opportunity with Orchard to help Turkish citizens access this groundbreaking gene therapy. We are committed to collaborating with physicians and the government to support the country’s eligible MLD patients in navigating a path to receive treatment with Libmeldy,” said Abidin Gülmüş, CEO of GEN.
About Libmeldy / OTL-200
Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.
The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.
For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the European Medicines Agency (EMA) website.
Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein, and Norway.
OTL-200 is an investigational therapy in the U.S.
Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
About Orchard
Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.
Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on Twitter and
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